CLE, Cutaneous Lupus Erythematosus (CLE), SLE, SLE (Systemic Lupus), Systemic Lupus Erythematosus
Conditions
Keywords
CLE, SLE, lupus, cutaneous lupus erythematosus, systemic lupus erythematosus, VENT-03, cGAS
Brief summary
The goal of this clinical trial is to learn if VENT-03 works to treat patients with cutaneous lupus erythematosus (CLE) who may or may not have systemic lupus erythematosus (SLE). Another goal is to learn about the safety of VENT-03 and how it is processed by the body. The main questions it aims to answer are: * Does VENT-03 affect the activity and severity of CLE? * What side effects do participants have when taking VENT-03? Researchers will compare VENT-03 to a placebo (a look-alike substance that contains no drug) to see if VENT-03 works to treat patients with CLE. Participants will: * Take VENT-03 or a placebo for 4 weeks, then all participants will switch to VENT-03 for another 8 weeks; * Visit the clinic once a month for checkups and tests.
Interventions
DRUGVENT-03
VENT-03 is a tablet
DRUGPlacebo
Placebo is a tablet
Sponsors
Ventus Therapeutics U.S., Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
Sponsor
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Cutaneous lupus: * CLASI-A score ≥8; * At least 1 active discoid lupus erythematosus (DLE) lesion, OR at least 1 active subacute CLE lesion * If participant has previous SLE diagnosis: * Positive antinuclear antibody test at Screening by immunofluorescent assay at the central laboratory with titer ≥ 1:80; * Meets the American College of Rheumatology/ European Alliance of Associations for Rheumatology 2019 criteria for SLE; and * Currently receiving at least one of the specified SLE medication treatments, at stable doses. Key
Exclusion criteria
* Meet protocol-specified infection or lab criteria; any other laboratory test results that, in the investigator's opinion, might place participant at unacceptable risk for participating in this study; * Moderate or severe liver impairment as classified by the Child-Pugh criteria (categories B and C); * Has drug-induced lupus, rather than 'idiopathic' lupus; * History of, or current, inflammatory joint or skin disease other than SLE and cutaneous lupus; * Diagnosis of select potentially confounding autoimmune disorders * Active severe or unstable neuropsychiatric SLE; * Hospitalization for a severe lupus flare in the past 3 months, or active severe SLE-driven disease, including lupus nephritis, for which in the opinion of the PI the protocol-specified SOC is insufficient; * History of or current diagnosis of anti-phospholipid syndrome; * History of any non-lupus disease that has required treatment with oral or parenteral corticosteroids for more than a total of 2 weeks within the last 24 weeks prior to Day 1; * Meets protocol specified medical history of infectious diseases and infections and/or opportunistic infection requiring hospitalization or parenteral antimicrobial treatment within specified timeframes; * Cancer screening results suspicious of malignancy or history of cancer within time specified with exceptions for curative therapy for squamous or basil cell carcinoma and cervical cancer in situ; and * Meets protocol specified exclusions related to concomitant medications.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Evaluate the effect of VENT-03 on the interferon gene signature in the skin | Baseline to End of Double-Blind Treatment (up to Day 28) | Percent change from baseline in interferon gene signature in the skin at Day 28 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Evaluate the effect of VENT-03 on CLE disease severity | Baseline to End of Double-Blind Treatment (up to Day 28) | Percent change from baseline in CLASI-A score at Day 28 |
| Evaluate effect of VENT-03 on CLE disease severity | Baseline to End of Double-Blind Treatment (up to Day 28) | Percentage of participants achieving CLASI-A 50 response (≥ 50% improvement in CLASI A score compared with baseline) at Day 28 |
| Change from Baseline in Myxovirus-Resistant Protein A (MXA) Immunostaining in Skin Biopsy | Baseline to End of Treatment (up to Day 84) | — |
| Number of participants with at least one Treatment Emergent Adverse Event (TEAE) and/or Serious Adverse Event (SAE) | Baseline to End of Treatment (up to Day 84) | An adverse event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. A TEAE is defined as an AE with an onset that occurs after receiving study drug. An SAE is an adverse event resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. |
| Number of participants with Moderate or Severe Treatment Emergent Adverse Events (TEAEs) | Baseline to End of Treatment (up to Day 84) | — |
| Percentage of Participants with ≥ 1 Treatment Emergent Adverse Event (AE) leading to Treatment Discontinuation | Baseline to End of Treatment (up to Day 84) | — |
| Cmax: Maximum Observed Plasma Concentration for VENT-03 | Day 1 pre-dose and at multiple time points (up to 6 hours) post-dose; Day 28 pre-dose and post-dose; Day 56 and Day 84 pre-dose | — |
| AUClast: Area Under the Plasma Concentration-Time Curve from Time 0 to the Time of the Last Quantifiable Concentration for VENT-03 | Day 1 pre-dose and at multiple time points (up to 6 hours) post-dose; Day 28 pre-dose and post-dose; Day 56 and Day 84 pre-dose | — |
Countries
Bulgaria, France, Georgia, Hungary, Poland, South Africa, Spain, United States
Contacts
CONTACTKrista Miller
Outcome results
None listed